AnatomyDiseasesChemicals and DrugsAnalytical, Diagnostic and Therapeutic Techniques and EquipmentPhenomena and ProcessesDisciplines and OccupationsAnthropology, Education, Sociology and Social PhenomenaInformation ScienceHealth Care
AzacitidineMyelodysplastic SyndromesAntimetabolites, AntineoplasticLeukemia, Myelomonocytic, ChronicAnemia, RefractoryAnemia, Refractory, with Excess of BlastsLeukemia, Myeloid, AcuteTreatment OutcomeLeukemia, MyeloidChromatography, Supercritical FluidThalidomideInjections, SubcutaneousCompassionate Use TrialsChromosomes, Human, Pair 5Anemia, SideroblasticInduction ChemotherapyConsolidation ChemotherapyDNA MethylationDNA Modification MethylasesBone MarrowMyelodysplastic-Myeloproliferative DiseasesMyeloproliferative DisordersDrug Administration ScheduleAcute DiseaseDrug ApprovalRemission InductionPreleukemiaBlood TransfusionCytarabinePrognosisSurvivalMonosomyChromosome AberrationsKaryotypingAntineoplastic Combined Chemotherapy ProtocolsHematopoietic Stem Cell TransplantationSurvival AnalysisTransplantation, HomologousAnemia, AplasticPancytopeniaChromosomes, Human, Pair 7International AgenciesRetrospective StudiesCytogenetic AnalysisEpigenomicsUnited States Food and Drug AdministrationBone Marrow CellsLeukemiaRecurrenceDisease ProgressionGranulocyte Precursor CellsChromosome DeletionMaximum Tolerated DoseNeoplasms, Second PrimaryBiological AvailabilityAntimetabolitesBone Marrow DiseasesIron OverloadAntineoplastic AgentsErythrocyte TransfusionTetrazolium SaltsHematologic AgentsTrisomyCytogeneticsHematopoietic Stem CellsAntigens, CD34Randomized Controlled Trials as TopicChelation TherapyErythropoietinInfusions, IntravenousFatigueSurvival RateEnzyme InhibitorsApoptosisHematopoiesisTransplantation ConditioningBlast CrisisThrombocytopeniaVidarabineBone Marrow ExaminationDrug Therapy, CombinationGranulocyte Colony-Stimulating FactorThrombocytosisAnemiaIsochromosomesHematologic DiseasesBlood Cell CountAnemia, MacrocyticCore Binding Factor Alpha 2 SubunitCell Line, TumorIn Situ Hybridization, FluorescencePrimary MyelofibrosisIron Chelating AgentsLeukemia, Myelomonocytic, AcuteChromosomes, Human, Pair 8Erythroid Precursor CellsFlow CytometryMutationChromosomes, Human, Pair 20Hematologic Neoplasms
SyndromeVenetoclaxDecitabineAcuteLower-Risk Myelodysplastic SyndromesHigher-risk myelodysplastic syndromesIntravenous or subcutaneous azacitidineTolerabilityHigh-risk myelodysplasticVIDAZASynergistic with azacitidineLenalidomideMyeloproliferativeMagrolimabPlaceboSafetyDoseMutationCombinationAllogeneicStudyClinicalONUREGPediatricInhibitorCancerSurvivalIntravenouslyEvaluateTreatmentDisease
Syndrome14
  • Two nucleoside inhibitors of DNA methylation, azacitidine and decitabine, are now standard of care for the treatment of the myelodysplastic syndrome, a deadly form of leukemia. (aacrjournals.org)
  • Several published literatures revealed that some oncogenes, such as MYC and MLL, have been identified to be amplified on dmins in AML and myelodysplastic syndrome (MDS) [ 6 ]. (hindawi.com)
  • 5-azacytidine (AZA) has become standard treatment for patients with higher-risk myelodysplastic syndrome (MDS). (biomedcentral.com)
  • In recent years, epigenetic therapy has become a treatment option for patients with higher-risk myelodysplastic syndrome (MDS) who are not considered candidates for intensive induction chemotherapy or allogeneic stem cell transplantation (SCT). (biomedcentral.com)
  • Myelodysplastic syndrome (MDS) refers to a heterogeneous group of closely related clonal hematopoietic disorders. (medscape.com)
  • Myelodysplastic syndrome (MDS) includes a group of clonal hematopoietic tumors, the main manifestations of which are cytopenia in the peripheral blood and dysplasia in the bone marrow. (clinlabdia.ru)
  • In addition, the article provides a generalized table showing the characteristic metabolic changes in various forms of myelodysplastic syndrome. (clinlabdia.ru)
  • Phase 1b evaluated INCB052793 plus standard therapy in relapsed/refractory multiple myeloma, acute myeloid leukemia (AML), or myelodysplastic syndrome (MDS). (elsevierpure.com)
  • Low-dose decitabine plus venetoclax is safe and effective as post-transplant maintenance therapy for high-risk acute myeloid leukemia and myelodysplastic syndrome. (devbrandcast.com)
  • Sabatolimab, an immunotherapeutic therapy targeting TIM-3 on immune and myeloid cells, was well-tolerated and led to durable clinical responses when combined with hypomethylating agents in patients with very high/high-risk myelodysplastic syndrome (vHR/HR-MDS) and newly diagnosed AML. (devbrandcast.com)
  • Efficacy and Safety of Sabatolimab (MBG453) in Combination with Hypomethylating Agents (HMAs) in Patients (Pts) with Very High/High-Risk Myelodysplastic Syndrome (vHR/HR-MDS) and Acute Myeloid Leukemia (AML): Final Analysis from a Phase Ib Study. (devbrandcast.com)
  • Most patients with myelodysplastic syndrome have greater than one mutation and a unique pattern of mutation with marked heterogeneity. (standardofcare.com)
  • Somatic mutation is not a static process in myelodysplastic syndrome and additional mutations will accumulate leading to more profound phenotypic worsening cytopenias, and approximately 30% of patients will eventually experience progression to secondary AML. (standardofcare.com)
  • The clinical trial aims to treat patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) with the drug azacitidine supplemented with vitamin C. (labroots.com)
Venetoclax9
  • Venetoclax with azacitidine (ven/aza) has emerged as a promising regimen for AML, with a high percentage of clinical remissions in newly diagnosed patients. (devbrandcast.com)
  • A combination regimen consisting of azacitidine with venetoclax and magrolimab showed clinical activity in older and high-risk patients with newly diagnosed AML. (devbrandcast.com)
  • Phase I/II Study of Azacitidine (AZA) with Venetoclax (VEN) and Magrolimab (Magro) in Patients (pts) with Newly Diagnosed Older/Unfit or High-Risk Acute Myeloid Leukemia (AML) and Relapsed/Refractory (R/R) AML. (devbrandcast.com)
  • In November 2021 , Curis initiated dosing in the combination stage of the Phase 1/2 study of CA-4948 plus azacitidine and CA-4948 plus venetoclax. (biospace.com)
  • The combination therapy portion includes two arms: CA-4948 plus azacitidine, for patients naïve to HMA, and CA-4948 plus venetoclax, for patients naïve to venetoclax. (biospace.com)
  • During anamnesis, lower urinary tract and gynecological cycles of decitabine, it was decided to switch AML therapy to surgeries, obstetrical history, neurological disorders, diabetes, azacitidine associated with venetoclax, without new reports and trauma were excluded as possible causes of urinary of urinary retention (Figure 1 ). (bvsalud.org)
  • A Phase I/II Study of Azacitidine, Venetoclax, and Gilteritinib for Patients With Acute Myeloid Leukemia or. (sparkcures.com)
  • A Phase I/II Study of Venetoclax in Combination With Azacitidine in Relapsed/Refractory High-Risk Myelodysp. (sparkcures.com)
  • It is evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of DSP107 as monotherapy and in combination with azacitidine or with azacitidine plus venetoclax in patients with relapsed/refractory AML or in MDS patients who have failed prior therapeutic regimens. (kahrbio.com)
Decitabine5
  • Currently, a handful of US Food and Drug Administration (FDA)-approved therapies for MDS are available, including lenalidomide, the erythroid maturation agent luspatercept, hypomethylating agents such as azacitidine and decitabine, and iron chelators such as deferasirox. (medscape.com)
  • However, the availability within the past decade of 3 drugs, azacitidine, lenalidomide, and decitabine, 3 - 5 which were approved by the FDA specifically for the treatment of MDS, has partly spurred the maturation rate of epidemiologic knowledge. (jnccn.org)
  • Currently, there are limited FDA-approved therapies for MDS, which include hypomethylating agents such as azacitidine and decitabine, iron chelators such as deferasirox, and lenalidomide. (medscape.com)
  • However, as the efficacy and safety of rational dosing regimens are lacking, we evaluated the effectiveness and safety of reduced-dose azacitidine (AZA) vs. decitabine (DAC) in adult MDS patients. (medscimonit.com)
  • Decitabine and cedazuridine combination is used to treat myelodysplastic syndromes (MDS), including chronic myelomonocytic leukemia (CMML), and certain types of anemia. (drugs.com)
Acute12
Lower-Risk Myelodysplastic Syndromes2
  • Thrice weekly azacitidine does not improve hematological responses in lower-risk myelodysplastic syndromes: a study of the Hoosier Oncology Group. (iu.edu)
  • Phase 1 study of JNJ-64619178, a protein arginine methyltransferase 5 inhibitor, in patients with lower-risk myelodysplastic syndromes. (cdc.gov)
Higher-risk myelodysplastic syndromes4
Intravenous or subcutaneous azacitidine5
  • Due to substantial differences in the pharmacokinetic parameters, the recommended dose and schedule for ONUREG ® are different from those for the intravenous or subcutaneous azacitidine products. (onuregpro.com)
  • Treatment of patients using intravenous or subcutaneous azacitidine at the recommended dosage of ONUREG ® may result in a fatal adverse reaction. (onuregpro.com)
  • Treatment with ONUREG ® at the doses recommended for intravenous or subcutaneous azacitidine may not be effective. (onuregpro.com)
  • Do not substitute ONUREG ® for intravenous or subcutaneous azacitidine. (onuregpro.com)
  • The indications and dosing regimen for ONUREG differ from that of intravenous or subcutaneous azacitidine ( 2.1 , 5.1 ). (nih.gov)
Tolerability4
  • Pierdomenico, F, Esteves, S & Almeida, A 2013, ' Efficacy and tolerability of 5-day azacytidine dose-intensified regimen in higher-risk MDS ', Annals of Hematology , vol. 92, no. 9, pp. 1201-1206. (ucp.pt)
  • Objective: Report final safety/tolerability and efficacy data. (ox.ac.uk)
  • When combined with azacitidine, CA-4948 will be dosed at 200 mg twice daily for 21 days of a 28-day cycle, followed by a 300 mg dose cohort if tolerability allows. (biospace.com)
  • A Phase Ib /II Open-label, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of APG-2575. (sparkcures.com)
High-risk myelodysplastic1
VIDAZA1
  • BOSTON, Aug 21 (Reuters) - Celgene Corp (CELG.O: Quote, Profile, Research, Stock Buzz) said on Thursday that U.S. regulators have given it the green light to add information to the label of its drug Vidaza showing it prolonged life in some patients with myelodysplastic syndromes, or MDS, a group of blood disorders that can lead to leukemia. (mds-foundation.org)
Synergistic with azacitidine2
  • Cluster of differentiation 47 blockade by magrolimab promotes macrophage-mediated phagocytosis of tumor cells and is synergistic with azacitidine, which increases expression of eat-me signals. (nih.gov)
  • Context: Magrolimab is a monoclonal antibody blocking CD47, a "don't eat me" signal overexpressed on cancer cells, which leads to tumor phagocytosis and is synergistic with azacitidine. (ox.ac.uk)
Lenalidomide2
Myeloproliferative1
  • Within myeloid neoplasms, chronic myelomonocytic leukemia (CMML) represents an attractive disease model since it is characterized by both myelodysplastic and myeloproliferative features, while retaining a relatively simple clonal composition 3 . (nature.com)
Magrolimab3
  • Magrolimab + azacitidine was well tolerated with promising efficacy in patients with untreated higher-risk MDS, including those with TP53 mutations. (nih.gov)
  • Design: Ph1b trial of magrolimab+azacitidine (NCT03248479). (ox.ac.uk)
  • Conclusions: Magrolimab+azacitidine was well tolerated, with promising efficacy in patients with untreated HR MDS, including those with TP53-mutated and -wild-type disease. (ox.ac.uk)
Placebo2
  • Azacitidine, placebo, and tamibarotene are the most frequent therapies in trials with RARA as an inclusion criteria [ 4 ]. (mycancergenome.org)
  • Only a clinical trial that combines azacitidine with the blinded addition of either vitamin C or a placebo will give the true answer as to whether or not vitamin C increases the efficacy of azacitidine in patients. (labroots.com)
Safety10
  • Our results demonstrate the safety and efficacy of a dose-intensified 5-day regimen. (ucp.pt)
  • The findings support future studies of assessing the safety and efficacy of the novel drug-chemotherapy combination as a maintenance strategy following stem cell transplantation across a range of blood cancers, according to the study authors. (curetoday.com)
  • Safety and efficacy have not been established. (drugs.com)
  • As we look ahead, we plan to provide a clinical data update in January from our ongoing clinical studies, including safety data from our Phase 1 study of CI-8993 and the latest safety and efficacy data from our CA-4948 study in AML/MDS patients with spliceosome or FLT3 mutations. (biospace.com)
  • Curis plans to provide an update on efficacy and safety from these patients in January. (biospace.com)
  • A Phase I Study Investigating the Safety & Efficacy of Danvatirsen as Monotherapy Followed by Combination W. (sparkcures.com)
  • The first part of the trial is a dose escalation study to explore the safety, efficacy, pharmacokinetic and pharmacodynamic profile of DSP107 when administered as monotherapy and in combination with azacitidine. (kahrbio.com)
  • cerning efficacy and safety of ribavirin are scarce ( 12 ). (cdc.gov)
  • Efficacy and safety of zanubrutinib plus R-CHOP in treatment of non-GCB DLBCL with extranodal involvement. (cdc.gov)
  • A Prospective Cohort Study Assessing the Relationship between Plasma Levels of Osimertinib and Treatment Efficacy and Safety. (cdc.gov)
Dose3
  • Due to patient and staffing constraints, we have treated 50 patients with a 5-day dose-intensified (500 mg/m2 total monthly dose divided in 5 days) azacitidine schedule in our center. (ucp.pt)
  • For pharmaceutical treatment, children need PK- and dose-finding studies, not separate proof of efficacy. (smw.ch)
  • Relationships of platelet glycoprotein specific antibody with therapeutic efficacy of short-term high-dose dexamethasone and bleeding score in the newly diagnosed adult patients with primary immune thrombocytopenia. (cdc.gov)
Mutation1
Combination2
  • Conclusion: Inhibition of JAK1 with INCB052793 (monotherapy or combination therapy) or itacitinib plus azacitidine did not demonstrate clinically meaningful responses in these patients with hematopoietic malignancies. (elsevierpure.com)
  • Efficacy of BRAF/MEK inhibitor combination highlights need for routine biomarker testing in pediatric brain cancer care. (aacrjournals.org)
Allogeneic1
  • The primary objective of this study is to demonstrate the efficacy of Iomab-B, in conjunction with a Reduced Intensity Conditioning (RIC) regimen and protocol-specified allogeneic hematopoietic stem cell transplant (HCT), versus Conventional Care. (mayo.edu)
Study2
  • Patients enrolled in the study were treated with APR-246 at 4500 mg intravenously from days 1 to 4 followed by azacitidine at 75mg/m2 from days 4 to 10 in 28-day cycles. (onclive.com)
  • The study was terminated for lack of efficacy. (elsevierpure.com)
Clinical1
ONUREG1
  • ONUREG ® is contraindicated in patients with known severe hypersensitivity to azacitidine or its components. (onuregpro.com)
Pediatric1
  • Most analyzed regulatory-demanded pediatric studies repeat(ed) proof of efficacy in adolescents and children and were/are medically of limited value. (smw.ch)
Inhibitor1
  • Phase 2 evaluated INCB052793 or itacitinib plus azacitidine in DNA methyltransferase inhibitor (DNMTi)-refractory AML or MDS. (elsevierpure.com)
Cancer1
  • Onconova Therapeutics, Inc. is a Phase 3-stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with an initial focus on Myelodysplastic Syndromes (MDS). (inceptua.com)
Survival2
  • Azacitidine has shown unprecedented survival advantage in these patients but its treatment schedule involves daily hospital administrations for 7 days every 4 weeks. (ucp.pt)
  • Survival efficacy of the PEGylated G-CSFs Maxy-G34 and neulasta in a mouse model of lethal H-ARS, and residual bone marrow damage in treated survivors. (iu.edu)
Intravenously2
  • Azacitidine 75 mg/m 2 was administered intravenously/subcutaneously once daily on days 1-7 of each 28-day cycle. (nih.gov)
  • Azacitidine 75 mg/m2 was administered intravenously/subcutaneously once daily on days 1-7 of each 28-day cycle. (ox.ac.uk)
Evaluate1
  • A041501, A Phase III Trial to Evaluate the Efficacy of the Addition of Inotuzumab Ozogamicin (a Conjugated Anti-CD22 Monoclonal Antibody) to Frontline Therapy in Young Adults (Ages 18-39 Years) With Newly Diagnosed Precursor B-Cell ALL Rochester, Minn. (mayo.edu)
Treatment5
  • Treatment recommendations for myelodysplastic syndromes (MDS) are based on a patient's prognostic grouping. (medscape.com)
  • 5-Azacytidine for the treatment of myelodysplastic syndromes. (aamds.org)
  • Treatment recommendations for myelodysplastic syndromes (MDS) are based on a patient's International Prognostic Scoring System (IPSS or IPSS-R) score in addition to evaluation of a patient's performance status. (medscape.com)
  • The most common reasons for treatment discontinuation were progressive disease (35.4% and 50.0%) and adverse events (22.9% and 20.0%) for INCB052793 and itacitinib plus azacitidine, respectively. (elsevierpure.com)
  • AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any drug, treatment or therapy listed on this website. (aamds.org)
Disease1
  • The incidence rate of the myelodysplastic syndromes (MDS) in the United States is approximately 3.4 per 100,000 people, accounting for more than 10,000 new diagnoses annually and an estimated 60,000 people living with the disease. (jnccn.org)